Efficient production of human FVIII in hemophilic mice using lentiviral vectors (Q45861949)

17 December 2017

title

Efficient production of human FVIII in hemophilic mice using lentiviral vectors (English)

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17 December 2017

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Neeltje A Kootstra

1

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17 December 2017

Ryusuke Matsumura

series ordinal

2

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17 December 2017

Inder M Verma

series ordinal

3

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17 December 2017

publication date

1 May 2003

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17 December 2017

published in

Molecular Therapy

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17 December 2017

volume

7

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17 December 2017

page(s)

623-631

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17 December 2017

issue

5 Pt 1

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17 December 2017

The molecular basis of hemophilia A in man.

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Ultrastructural localization of factor VIII procoagulant antigen in human liver hepatocytes

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7 January 2021

Toward gene therapy for hemophilia A: long-term persistence of factor VIII-secreting fibroblasts after transplantation into immunodeficient mice.

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In vivo production of human factor VII in mice after intrasplenic implantation of primary fibroblasts transfected by receptor-mediated, adenovirus-augmented gene delivery

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Gene therapy for hemophilia A: production of therapeutic levels of human factor VIII in vivo in mice

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7 January 2021

Long-term persistence of human bone marrow stromal cells transduced with factor VIII-retroviral vectors and transient production of therapeutic levels of human factor VIII in nonmyeloablated immunodeficient mice

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Genetic induction of immune tolerance to human clotting factor VIII in a mouse model for hemophilia A

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Vesicular stomatitis virus G glycoprotein pseudotyped retroviral vectors: concentration to very high titer and efficient gene transfer into mammalian and nonmammalian cells

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Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice

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Short-term correction of factor VIII deficiency in a murine model of hemophilia A after delivery of adenovirus murine factor VIII in utero

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Partial correction of murine hemophilia A with neo-antigenic murine factor VIII.

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Systemic delivery of an adenoviral vector encoding canine factor VIII results in short-term phenotypic correction, inhibitor development, and biphasic liver toxicity in hemophilia A dogs.

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Coexpression of factor VIII heavy and light chain adeno-associated viral vectors produces biologically active protein

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Development of a self-inactivating lentivirus vector.

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In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector

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Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences

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Expression vector system based on the chicken beta-actin promoter directs efficient production of interleukin-5.

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Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A.

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Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors

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Transduction of human CD34+ cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors

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7 January 2021

Induction of tolerance to human factor VIII in mice

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7 January 2021

Use of a liver-specific promoter reduces immune response to the transgene in adenoviral vectors

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Lentiviral vectors containing the human immunodeficiency virus type-1 central polypurine tract can efficiently transduce nondividing hepatocytes and antigen-presenting cells in vivo

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In vitro selection of lentivirus vector-transduced human CD34+ cells

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Identifiers

DOI

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17 December 2017

PubMed ID

12718905

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17 December 2017